IP Library Granted Patent US 9,943,565
Granted Patent B2
US 9,943,565 · App. 14/959,470 · Granted Apr 17, 2018

Methods and compositions for treating trinucleotide repeat disorders

Inventors: Philip D. Gregory (Orinda, CA); Edward J. Rebar (San Francisco, CA); H. Steve Zhang (Richmond, CA)
Assignee: Sangamo Therapeutics, Inc.
A61K38/1709C07K14/47C12N15/907A61K38/00A61K48/00C07K2319/00C07K2319/71C07K2319/81
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Quick Facts
Patent No.
US 9,943,565
App. No.
14/959,470
Granted
Apr 17, 2018
Kind
B2
Abstract

Disclosed herein are methods and compositions for treating trinucleotide repeat disorders.

Claims (8)

1. A cell comprising a complex of a non-naturally occurring zinc finger protein transcription factor and a target site in an Htt gene, wherein the zinc finger protein transcription factor comprises a transcriptional repression domain and a finger protein comprising 5 or 6 zinc finger recognition regions, ordered F1 to F5 or F1 to F6 from N-terminus to C-terminus, wherein the zinc finger protein comprises the recognition helix regions in the order shown in a single row of Table 1 bound to the target site in the Htt gene, the target site comprising sequences as set forth in any of SEQ ID NO:20-23 and 36-39 and further wherein Htt gene expression is repressed in the cell.

2. The cell of claim 1 , wherein the Htt gene is wild-type.

3. The cell of claim 2 , wherein the zinc finger protein binds to a SNP associated with a wild-type allele.

4. The cell of claim 1 , wherein the Htt gene comprises one or more expanded trinucleotide repeats.

5. The cell of claim 4 , wherein the expanded trinucleotide repeat encodes poly-glutamine.

6. The cell of claim 4 , wherein the expanded trinucleotide repeat encodes poly-serine.

7. The cell of claim 4 , wherein the zinc finger protein binds to a SNP associated with the expanded trinucleotide repeat.

8. The cell of claim 1 , wherein the cell comprises a multimer of the zinc finger protein transcription factors and wherein the multimer preferentially binds to expanded trinucleotide tracts as compared to wild-type trinucleotide repeat tracts.

Assignments (1)
CHANGE OF NAME Recorded Jan 18, 2018
From: SANGAMO BIOSCIENCES, INC.
To: SANGAMO THERAPEUTICS, INC.
Reel/Frame 045090/0516 →
Continuity (4)
Continuation 12804764 · Jul 28, 2010
Provisional Application 61271913 · Jul 28, 2009
Provisional Application 61273009 · Jul 29, 2009
Related Publication 20160082081A1 · Mar 24, 2016